ArticleFrontiers in medicine2026
Clinical safety and tolerability of
Article in Frontiers in medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Authors and funding
7 authors.
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Abstract
Background: ATTR amyloidosis is a disease caused by abnormal deposition of TTR (transthyretin) protein in tissues. ART001 is an Methods: In an investigator-initiated trial (IIT) of ART001 for 10 ATTR Amyloidosis patients, each patient was given one dose of ART001 which ranged from 0.05 mg/kg to 1.0 mg/kg. The aim was to evaluate ART001's safety, side effects, PK, PD, and efficacy based on circulating TTR protein levels. Results: At 0.7 mg/kg in 3 subjects and 1 mg/kg in 3 subjects, TTR protein reductions averaged 84 and 92% at 72 weeks. No infusion-related reactions (IRRs), serious adverse events (SAEs) or serious adverse reactions (SARs) were observed. Conclusion: A single injection of ART001 achieved > 80% TTR knock-down at doses > 0.5 mg/kg and lasted for at least 72 weeks without IRRs, SARs or SAEs. ART001 has the potential to be a safe, effective and permanent therapeutic option for ATTR Amyloidosis patients. (Funded by Accuredit Therapeutics). Clinical trial registration: Trial registration: ChiCTR, ChiCTR2400081216. Registered 26
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.